{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,3]],"date-time":"2026-06-03T07:38:44Z","timestamp":1780472324121,"version":"3.54.1"},"reference-count":36,"publisher":"Wiley","issue":"9","license":[{"start":{"date-parts":[[2021,7,31]],"date-time":"2021-07-31T00:00:00Z","timestamp":1627689600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100012041","name":"Versus Arthritis","doi-asserted-by":"publisher","award":["SR\/20094"],"award-info":[{"award-number":["SR\/20094"]}],"id":[{"id":"10.13039\/501100012041","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100001279","name":"Great Ormond Street Hospital Charity","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100001279","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100010876","name":"Vasculitis UK","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100010876","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["acrjournals.onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Arthritis &amp; Rheumatology"],"published-print":{"date-parts":[[2021,9]]},"abstract":"<jats:sec><jats:title>Objective<\/jats:title><jats:p>Cyclophosphamide (CYC) is used in clinical practice off\u2010label for the induction of remission in childhood polyarteritis nodosa (PAN). Mycophenolate mofetil (MMF) might offer a less toxic alternative. This study was undertaken to explore the relative effectiveness of CYC and MMF treatment in a randomized controlled trial (RCT).<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>This was an international, open\u2010label, Bayesian RCT to investigate the relative effectiveness of CYC and MMF for remission induction in childhood PAN. Eleven patients with newly diagnosed childhood PAN were randomized (1:1) to receive MMF or intravenous CYC; all patients received the same glucocorticoid regimen. The primary end point was remission within 6 months while compliant with glucocorticoid taper. Bayesian distributions for remission rates were established a priori for MMF and CYC by experienced clinicians and updated to posterior distributions on trial completion.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Baseline disease activity and features were similar between the 2 treatment groups. The primary end point was met in 4 of 6 patients (67%) in the MMF group and 4 of 5 patients (80%) in the CYC group. Time to remission was shorter in the MMF group compared to the CYC group (median 7.1 weeks versus 17.6 weeks). No relapses occurred in either group within 18 months. Two serious infections were found to be likely linked to MMF treatment. Physical and psychosocial quality\u2010of\u2010life scores were superior in the MMF group compared to the CYC group at 6 months and 18 months. Combining the prior expert opinion with results from the present study provided posterior estimates of remission of 71% for MMF (90% credibility interval [90% CrI] 51, 83) and 75% for CYC (90% CrI 57, 86).<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>The present results, taken together with prior opinion, indicate that rates of remission induction in childhood PAN are similar with MMF treatment and CYC treatment, and MMF treatment might be associated with better health\u2010related quality of life than CYC treatment.<\/jats:p><\/jats:sec>","DOI":"10.1002\/art.41730","type":"journal-article","created":{"date-parts":[[2021,3,24]],"date-time":"2021-03-24T07:30:51Z","timestamp":1616571051000},"page":"1673-1682","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":24,"title":["Mycophenolate Mofetil Versus Cyclophosphamide for Remission Induction in Childhood Polyarteritis Nodosa: An Open\u2010Label, Randomized, Bayesian Noninferiority Trial"],"prefix":"10.1002","volume":"73","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-6178-6893","authenticated-orcid":false,"given":"Paul A.","family":"Brogan","sequence":"first","affiliation":[{"name":"University College London Great Ormond Street Institute of Child Health and Great Ormond Street Hospital for Children NHS Foundation Trust  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Barbara","family":"Arch","sequence":"additional","affiliation":[{"name":"University of Liverpool  Liverpool UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Helen","family":"Hickey","sequence":"additional","affiliation":[{"name":"University of Liverpool  Liverpool UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8792-4219","authenticated-orcid":false,"given":"Jordi","family":"Anton","sequence":"additional","affiliation":[{"name":"St. Joan de D\u00e9u Hospital  Barcelona Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Este","family":"Iglesias","sequence":"additional","affiliation":[{"name":"St. Joan de D\u00e9u Hospital  Barcelona Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eileen","family":"Baildam","sequence":"additional","affiliation":[{"name":"Alder Hey Children\u2019s NHS Foundation Trust  Liverpool UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kamran","family":"Mahmood","sequence":"additional","affiliation":[{"name":"Alder Hey Children\u2019s NHS Foundation Trust  Liverpool UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gavin","family":"Cleary","sequence":"additional","affiliation":[{"name":"Alder Hey Children\u2019s NHS Foundation Trust  Liverpool UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Elena","family":"Moraitis","sequence":"additional","affiliation":[{"name":"Great Ormond Street Hospital for Children NHS Foundation Trust  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Charalampia","family":"Papadopoulou","sequence":"additional","affiliation":[{"name":"Great Ormond Street Hospital for Children NHS Foundation Trust  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael W.","family":"Beresford","sequence":"additional","affiliation":[{"name":"University of Liverpool  Liverpool UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Phil","family":"Riley","sequence":"additional","affiliation":[{"name":"Royal Manchester Children\u2019s Hospital  Manchester UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Selcan","family":"Demir","sequence":"additional","affiliation":[{"name":"Hacettepe University  Ankara Turkey"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2883-7868","authenticated-orcid":false,"given":"Seza","family":"Ozen","sequence":"additional","affiliation":[{"name":"Hacettepe University  Ankara Turkey"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Giovanna","family":"Culeddu","sequence":"additional","affiliation":[{"name":"Bangor University  Bangor UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dyfrig A.","family":"Hughes","sequence":"additional","affiliation":[{"name":"Bangor University  Bangor UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pavla","family":"Dolezalova","sequence":"additional","affiliation":[{"name":"General University Hospital in Prague and Charles University  Prague Czech Republic"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lisa\u00a0V.","family":"Hampson","sequence":"additional","affiliation":[{"name":"Lancaster University  Lancaster UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"John","family":"Whitehead","sequence":"additional","affiliation":[{"name":"Lancaster University  Lancaster UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"David","family":"Jayne","sequence":"additional","affiliation":[{"name":"University of Cambridge  Cambridge UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nicola","family":"Ruperto","sequence":"additional","affiliation":[{"name":"Instituto Giannina Gaslini IRCCS UOSID Centro Trial  Genoa Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Catrin","family":"Tudur\u2010Smith","sequence":"additional","affiliation":[{"name":"University of Liverpool  Liverpool UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Despina","family":"Eleftheriou","sequence":"additional","affiliation":[{"name":"University College London Great Ormond Street Institute of Child Health and Great Ormond Street Hospital for Children NHS Foundation Trust  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2021,7,31]]},"reference":[{"key":"e_1_2_7_2_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00467-009-1336-1"},{"key":"e_1_2_7_3_1","doi-asserted-by":"publisher","DOI":"10.1136\/ard.2009.116657"},{"key":"e_1_2_7_4_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(02)11279-7"},{"key":"e_1_2_7_5_1","doi-asserted-by":"publisher","DOI":"10.1002\/art.38024"},{"key":"e_1_2_7_6_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jpeds.2004.06.046"},{"key":"e_1_2_7_7_1","doi-asserted-by":"publisher","DOI":"10.1046\/j.1365-2249.2003.02081.x"},{"issue":"2","key":"e_1_2_7_8_1","first-page":"Sii168","article-title":"Polyarteritis nodosa in children","volume":"69","author":"Brogan PA","year":"2002","journal-title":"Clev Clin J Med"},{"key":"e_1_2_7_9_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1307362"},{"key":"e_1_2_7_10_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1307361"},{"key":"e_1_2_7_11_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMc1801927"},{"key":"e_1_2_7_12_1","doi-asserted-by":"publisher","DOI":"10.1002\/art.39699"},{"key":"e_1_2_7_13_1","doi-asserted-by":"publisher","DOI":"10.1093\/rheumatology\/key322"},{"key":"e_1_2_7_14_1","first-page":"537","article-title":"The effect of cortisone on the lesions of periarteritis nodosa","volume":"27","author":"Baggenstoss AH","year":"1951","journal-title":"Am J Pathol"},{"key":"e_1_2_7_15_1","doi-asserted-by":"publisher","DOI":"10.1093\/rheumatology\/36.12.1290"},{"key":"e_1_2_7_16_1","doi-asserted-by":"publisher","DOI":"10.1002\/art.1780381116"},{"key":"e_1_2_7_17_1","doi-asserted-by":"publisher","DOI":"10.1002\/art.10922"},{"key":"e_1_2_7_18_1","doi-asserted-by":"publisher","DOI":"10.1002\/art.27340"},{"key":"e_1_2_7_19_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2249.2011.04364.x"},{"key":"e_1_2_7_20_1","doi-asserted-by":"publisher","DOI":"10.1681\/ASN.2008101028"},{"key":"e_1_2_7_21_1","doi-asserted-by":"publisher","DOI":"10.1136\/annrheumdis-2018-214245"},{"key":"e_1_2_7_22_1","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0120981"},{"key":"e_1_2_7_23_1","doi-asserted-by":"publisher","DOI":"10.1136\/adc.2010.188946"},{"key":"e_1_2_7_24_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2125.2012.04305.x"},{"key":"e_1_2_7_25_1","doi-asserted-by":"publisher","DOI":"10.1136\/ard.2009.116624"},{"key":"e_1_2_7_26_1","doi-asserted-by":"publisher","DOI":"10.1136\/annrheumdis-2012-202111"},{"key":"e_1_2_7_27_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.lpm.2013.02.067"},{"key":"e_1_2_7_28_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13075-015-0545-1"},{"key":"e_1_2_7_29_1","doi-asserted-by":"publisher","DOI":"10.1093\/rheumatology\/kew029"},{"issue":"23","key":"e_1_2_7_30_1","first-page":"S1","article-title":"Cross\u2010cultural adaptation and psychometric evaluation of the Childhood Health Assessment Questionnaire (CHAQ) and the Child Health Questionnaire (CHQ) in 32 countries: review of the general methodology","volume":"19","author":"Ruperto N","year":"2001","journal-title":"Clin Exp Rheumatol"},{"key":"e_1_2_7_31_1","doi-asserted-by":"publisher","DOI":"10.2165\/11599120-000000000-00000"},{"key":"e_1_2_7_32_1","doi-asserted-by":"publisher","DOI":"10.1016\/0168-8510(96)00822-6"},{"key":"e_1_2_7_33_1","doi-asserted-by":"publisher","DOI":"10.1002\/sim.6225"},{"key":"e_1_2_7_34_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(10)60413-8"},{"key":"e_1_2_7_35_1","doi-asserted-by":"publisher","DOI":"10.1002\/hec.635"},{"key":"e_1_2_7_36_1","doi-asserted-by":"publisher","DOI":"10.1136\/ard.2009.116657"},{"key":"e_1_2_7_37_1","doi-asserted-by":"publisher","DOI":"10.1136\/annrheumdis-2016-210002"}],"container-title":["Arthritis &amp; Rheumatology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/art.41730","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1002\/art.41730","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/acrjournals.onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/art.41730","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,10,8]],"date-time":"2025-10-08T16:17:52Z","timestamp":1759940272000},"score":1,"resource":{"primary":{"URL":"https:\/\/acrjournals.onlinelibrary.wiley.com\/doi\/10.1002\/art.41730"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2021,7,31]]},"references-count":36,"journal-issue":{"issue":"9","published-print":{"date-parts":[[2021,9]]}},"alternative-id":["10.1002\/art.41730"],"URL":"https:\/\/doi.org\/10.1002\/art.41730","archive":["Portico"],"relation":{},"ISSN":["2326-5191","2326-5205"],"issn-type":[{"value":"2326-5191","type":"print"},{"value":"2326-5205","type":"electronic"}],"subject":[],"published":{"date-parts":[[2021,7,31]]},"assertion":[{"value":"2020-07-09","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2021-03-11","order":2,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2021-07-31","order":3,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}